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Eltrombopag in chronic myelomonocytic leukemia with severe thrombocytopenia. A Groupe Francophone des Myélodysplasies (GFM) study
Leukemia ( IF 12.8 ) Pub Date : 2024-09-12 , DOI: 10.1038/s41375-024-02402-8
Florence Rabian 1 , Sylvie Chevret 2 , Bérengère Gruson 3 , Sylvain Thépot 4 , Anouk Walter-Petrich 2 , Thorsten Braun 5 , Norbert Vey 6 , José Miguel Torregrosa-Diaz 7 , Pierre Peterlin 8 , Andrea Toma 9 , Maud D'Aveni 10 , Jacques Delaunay 11 , Laurence Legros 12 , Nathalie Droin 13 , Fatiha Chermat 14 , Daniel Lusina 15 , Lionel Adès 1, 16 , Rosa Sapena 14 , Eric Solary 13 , Pierre Fenaux 1, 16 , Raphael Itzykson 1, 16
Affiliation  

Severe thrombocytopenia (<50 ×109/L) occurs in 10–30% of Chronic Myelomonocytic Leukemias (CMML) and leads to significant mortality through increased risk of bleeding and disease progression [1]. Its management is ill codified [2].

The thrombopoietin receptor agonist Eltrombopag improves platelet counts in lower-risk myelodysplastic syndromes [3]. Its activity in CMML has been investigated in retrospective case series of up to eleven patients [4,5,6], or as part of a phase I MDS trial [7].



中文翻译:


艾曲波帕治疗伴有严重血小板减少症的慢性粒单核细胞白血病。Groupe Francophone des Myélodysplasies (GFM) 研究



10-30% 的慢性粒单核细胞白血病 (CMML) 会出现严重的血小板减少症 (<50 ×109/L),并通过增加出血和疾病进展的风险导致大量死亡 [1]。其管理没有编纂成文[2]。


血小板生成素受体激动剂艾曲波帕可改善低危骨髓增生异常综合征的血小板计数 [3]。已在多达 11 例患者的回顾性病例系列研究中研究了其在 CMML 中的活性 [4,5,6],或作为 I 期 MDS 试验的一部分 [7]。

更新日期:2024-09-12
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